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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young AdultsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Ewing sarcoma, Eye Diseases, Hereditary, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Malignant Peripheral Nerve Sheath Tumors, Myosarcoma, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, benign urinary system neoplasm, carcinoma, clear cell sarcoma, connective tissue neoplasm, desmoplastic small round cell tumor, epithelial neoplasm, eye disorder, fibrosarcoma, hepatoblastoma, hereditary disease, hereditary neoplastic syndrome, kidney cancer, kidney disorder, mixed neoplasm, myomatous neoplasm, neoplasm, nerve sheath neoplasm, nervous system disorder, neuroblastoma, neuroepithelial neoplasm, neurofibroma, neurofibrosarcoma, neuromuscular disease, ocular cancer, osteosarcoma, peripheral nervous system disorder, peripheral nervous system neoplasm, primitive neuroectodermal tumor, retinal cancer, retinal disorder, retinoblastoma, rhabdoid tumor, rhabdomyosarcoma, sarcoma, synovial sarcoma, urinary system disorderActive, not recruitingPhase 12026-10-08NCT03618381
AZD6244 Hydrogen Sulfate for Children With Nervous System TumorsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaActive, not recruitingPhase 1 / Phase 22026-10-08NCT01362803
Natural History Study of Patients With Neurofibromatosis Type ICongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, connective tissue neoplasm, cranial nerve neoplasm, cranial nerve neuropathy, epithelial neoplasm, eye disorder, fibrosarcoma, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neurofibrosarcoma, neuromuscular disease, optic nerve disorder, optic nerve glioma, optic nerve neoplasm, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma, primitive neuroectodermal tumor, sarcomaActive, not recruitingNot stated2026-10-08NCT00924196
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform NeurofibromasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaActive, not recruitingPhase 22026-10-06NCT03962543
Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade GliomaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, astrocytoma (excluding glioblastoma), epithelial neoplasm, glioblastoma, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, primitive neuroectodermal tumorNot yet recruitingPhase 22026-10-05NCT07521657
Study of Cabozantinib With Selumetinib for Plexiform NeurofibromasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaRecruitingPhase 12026-10-02NCT06502171
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaRecruitingNot stated2026-10-02NCT02544022
Natural History Study of Patients With Neurofibromatosis Type 2Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ear Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Otorhinolaryngologic Neoplasms, acoustic neuroma, central nervous system cancer, central nervous system disorder, cranial nerve neoplasm, cranial nerve neuropathy, ependymoma, epithelial neoplasm, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, meningioma, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroendocrine neoplasm, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neuroma, otorhinolaryngologic disease, peripheral nervous system neoplasm, primitive neuroectodermal tumor, retrocochlear disease, schwannoma, spinal cord disorder, spinal cord neoplasm, tumor of meninges, vascular cancer, vestibulocochlear nerve disorderRecruitingNot stated2026-10-02NCT00598351
Neurocutaneous Syndromes in Children: Prevalence and Clinical CharacteristicsAbnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Malformations of Cortical Development, Group I, Neoplasms by Histologic Type, Neoplasms, Multiple Primary, Neoplasms, Nerve Tissue, Skin Abnormalities, Skin and Connective Tissue Diseases, Sturge-Weber syndrome, angiomatosis, cardiovascular disorder, central nervous system malformation, cerebral cortical dysplasia, ectodermal dysplasia syndrome, hamartoma, hemangioma, hereditary disease, hereditary neoplastic syndrome, hereditary skin disorder, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, skin disorder, tuberous sclerosis, vascular cancer, vascular disorderNot yet recruitingNot stated2026-09-28NCT07843823
Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosisRecruitingNot stated2026-09-25NCT07088991
Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNsNeoplasms by Histologic Type, Neoplasms, Nerve Tissue, neoplasm, nerve sheath neoplasm, nervous system disorder, neurofibroma, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaActive, not recruitingNot stated2026-09-23NCT06175637
NF-1, Nutraceutical InterventionCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorderActive, not recruitingPhase 12026-09-23NCT05363267
Metarrestin (ML-246) in Subjects With Metastatic Solid TumorsDigestive System Diseases, Digestive System Neoplasms, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Nerve Tissue, colonic disorder, colorectal neoplasm, connective tissue neoplasm, digestive system disorder, digestive system neoplasm, endocrine system disorder, fibrosarcoma, intestinal disorder, malignant endocrine neoplasm, neoplasm, nerve sheath neoplasm, nervous system disorder, neurofibroma, neurofibrosarcoma, neuromuscular disease, pancreas disorder, pancreatic neoplasm, peripheral nervous system disorder, peripheral nervous system neoplasm, rectal disorder, sarcomaRecruitingPhase 12026-09-23NCT04222413
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade GliomaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, astrocytoma (excluding glioblastoma), epithelial neoplasm, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, low grade glioma, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, primitive neuroectodermal tumorActive, not recruitingPhase 1 / Phase 22026-09-21NCT01089101
DoD Award NF230020 Identification of Metabolic Markers and Statistical Prediction of MPNST for Rapid Diagnosis and Assessment of Surgical MarginsNeoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Nerve Tissue, connective tissue neoplasm, fibrosarcoma, neoplasm, nerve sheath neoplasm, nervous system disorder, neurofibroma, neurofibrosarcoma, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, sarcomaRecruitingNot stated2026-09-18NCT07298083
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical NeurofibromasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorderRecruitingPhase 1 / Phase 22026-09-16NCT04750928
MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform NeurofibromasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaActive, not recruitingPhase 22026-09-15NCT02407405
How Can the Experience of Hospital Outpatient Departments be Improved?Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ear Diseases, NF2-related schwannomatosis, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, Neurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, communication disorder, eye disorder, hearing disorder, hearing loss disorder, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, learning disability, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurodevelopmental disorder, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, otorhinolaryngologic disease, peripheral nervous system disorder, psychiatric disorder, vision disorderNot yet recruitingNot stated2026-09-14NCT07818213
Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorderRecruitingNot stated2026-09-11NCT07221331
Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibromaEnrolling by invitationNot stated2026-09-09NCT05238909
Safety and Tolerability of TNG462 in Patients With MTAP-deleted Solid TumorsNeoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Nerve Tissue, adenocarcinoma, adenoma, carcinoma, cholangiocarcinoma, connective tissue neoplasm, epithelial neoplasm, fibrosarcoma, mesothelial neoplasm, mesothelioma, neoplasm, nerve sheath neoplasm, nervous system disorder, neurofibroma, neurofibrosarcoma, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, sarcomaRecruitingPhase 1 / Phase 22026-09-08NCT05732831
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic LeukemiaCBL-related disorder, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, bone marrow disorder, hematologic disorder, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, juvenile myelomonocytic leukemia, leukemia, myelodysplastic/myeloproliferative disease, myeloid leukemia, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorderRecruitingPhase 1 / Phase 22026-09-04NCT05849662
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Histologic Type, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Nerve Tissue, connective tissue neoplasm, fibrosarcoma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neurofibrosarcoma, neuromuscular disease, peripheral nervous system disorder, peripheral nervous system neoplasm, plexiform neurofibroma, sarcomaRecruitingNot stated2026-09-02NCT06222203
Phase 1 Study of MRTX1719 in Solid Tumors With MTAP DeletionDigestive System Diseases, Digestive System Neoplasms, Malignant Peripheral Nerve Sheath Tumors, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Nerve Tissue, Thoracic Neoplasms, adenoma, bronchial neoplasm, bronchogenic carcinoma, connective tissue neoplasm, endocrine system disorder, epithelial neoplasm, fibrosarcoma, lung disorder, lung neoplasm, malignant endocrine neoplasm, mesothelial neoplasm, mesothelioma, neoplasm, nerve sheath neoplasm, nervous system disorder, neurofibroma, neurofibrosarcoma, neuromuscular disease, non-small cell lung carcinoma, pancreas disorder, pancreatic adenocarcinoma, pancreatic neoplasm, peripheral nervous system disorder, peripheral nervous system neoplasm, respiratory system disorder, respiratory tract neoplasm, sarcomaRecruitingPhase 12026-09-02NCT05245500
Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Ear Diseases, NF2-related schwannomatosis, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Otorhinolaryngologic Neoplasms, acoustic neuroma, central nervous system cancer, cranial nerve neoplasm, cranial nerve neuropathy, ependymoma, epithelial neoplasm, glioma, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, meningioma, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neuroendocrine neoplasm, neuroepithelial neoplasm, neurofibroma, neurofibromatosis, neuroma, otorhinolaryngologic disease, peripheral nervous system neoplasm, primitive neuroectodermal tumor, retrocochlear disease, schwannoma, tumor of meninges, vascular cancer, vestibulocochlear nerve disorderRecruitingPhase 22026-09-02NCT04374305